News Regeneron drug on track to be second FOP treatment There is only one approved drug for ultra-rare disease FOP on the market, but Regeneron is hoping to add a second.
News Servier bets $450m on Fragile X syndrome drug Servier has pushed its emerging neurology pipeline into the clinical trials stage by licensing a drug for Fragile X syndrome from the UK's Kaerus.
News Sanofi breaks new ground for BTK drugs with ITP approval Sanofi's BTK inhibitor Wayrilz has become the first drug in the class to be approved for rare platelet-busting disease immune thrombocytopenia.
News Ionis brings long-acting treatment option to HAE patients Ionis gets FDA nod for its HAE attack prophylactic treatment Dawnzera, offering less frequent dosing than its rivals.
News PTC stung by FDA decision not to approve rare disease drug It looks like PTC will have to carry out another trial of its drug for Friedreich ataxia before the FDA will consider approval.
News Novartis taps Atropos AI for earlier PNH diagnoses Novartis teams up with real-world evidence specialist Atropos Health, hoping to speed up the diagnosis and treatment of rare disease PNH.
